On May 20th, the House Energy and Commerce Subcommittee on Health held a hearing entitled, 21st Century Cures: The President’s Council of Advisors on Science and Technology (PCAST) Report on Drug Innovation.

The hearing was led by Chairman Joe Pitts (R-PA) and is part of the new 21st Century Cures initiative that seeks to accelerate the discovery, development and delivery of promising new treatments to patients.…

In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profile in twelve boys with Duchenne. Plasma levels of the drug were variable and the company speculates that diet and the course of the…

The Internal Revenue Service (IRS) recently reversed a policy and now allows parents of people with disabilities who receive Medicaid Home and Community Based waiver funds, for care giving services provided to their children, to exclude those funds from their incomes. Notice 2014-7 describes changes in the IRS policy. These changes may apply to foster and biological parents caring for a person with Duchenne muscular…

Originally signed into law in 2001, the MD-CARE Act has birthed key discoveries in the muscular dystrophies, including the scientific justification for why exon skipping therapies could potentially work in humans.

A recent PPMD-funded study at Cedars Sinai Medical Center in Los Angeles (published in the May 7, 2014, online issue of Neurology®) demonstrates that those with Duchenne have impaired circulation in their muscles, which may contribute to muscle weakness, and that the approved vasodilator tadalafil can restore that…

This past year has brought many new and exciting changes to DuchenneConnect, the robust and cutting-edge registry and resource that connects Duchenne and Becker patients with actively recruiting clinical trials and research studies, and educates patients and families about Duchenne and Becker research. The registry, which was established in 2007, has matured beyond a standard registry and into an innovative…